RARE Daily

Sanofi Hands Sickle Cell Disease Program Back to Sangamo

January 6, 2022

Sangamo Therapeutics reported that Sanofi is terminating their collaboration related to SAR445136, a zinc finger nuclease gene-edited cell therapy candidate for the treatment of sickle cell disease and handing all rights and obligations in development back to Sangamo.

Photo: Sandy Macrae, CEO of Sangamo

Sangamo said Sanofi’s decision to change its cell therapy strategy to allogeneic cell therapies led to the termination of their global research and license agreement to develop genomic medicines for hemoglobinopathies. The transition is expected to be completed by mid-2022.

The termination comes just one month after the companies reported preliminary proof-of-concept data at ASH 2021 showing no treatment-related adverse events and that treated patients did not require blood transfusions post engraftment. The data reported was through 91 weeks for the longest-treated patient and through 26 weeks for the most recently treated patient. All four treated patients experienced increases in total hemoglobin, fetal hemoglobin, and percent F cells and none required blood transfusions post engraftment. Total hemoglobin stabilized by Week 26 after treatment with SAR445136 in all four patients.

“Although the preliminary phase 1/2 clinical data for the autologous sickle cell treatment are encouraging, Sanofi has made the decision to terminate the collaboration on the SAR445136 program, which is consistent with our strategy to focus on universal genomic medicine approaches,” said John Reed, global head of research and development at Sanofi. “Sangamo has been a good partner and this decision is not a reflection on the potential of the SAR445136 program. We continue to view them as a pioneer in the area of genomic medicines and will explore other possible collaboration opportunities as we work together to transition the autologous sickle cell program back to Sangamo.”

Sangamo remains focused on advancing the program, including seeking a new collaboration partner. The company says the phase 1/2 PRECIZN-1 study of investigational SAR445136 is expected to be completed as planned with the final patients in study expected to be dosed in third quarter of 2022 and expects that Sanofi will continue to pay the costs of the study until the termination of their deal on June 28, 2022.

People with sickle cell disease have a mutation that alters hemoglobin, the protein in red blood cells that carries oxygen to cells throughout the body. The sickle mutation causes red blood cells to have an abnormal sickle or crescent shape, which makes them inefficient in their oxygen-carrying capacity and leads to chronic anemia, vaso-occlusive crises with severe pain, multi-organ damage, complications like stroke, and a shortened life expectancy. Globally, 300,000 people are born with sickle cell disease every year, and approximately 100,000 people are living with sickle cell disease in the United States.

“We remain committed to progressing this program and believe SAR445136 has the potential to relieve people living with sickle cell disease of some of their most challenging symptoms,” said Sandy Macrae, CEO of Sangamo. He said the company expected to complete the phase 1/2 study as planned and will explore alternatives for moving the program forward.

Author: Rare Daily Staff

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